Pioneering Solutions in Rare Disease Research:
MLM Medical Labs’ Customized Preclinical Models

by Katie Dineen, B.S., Business Development Manager Preclinical Laboratory Services 

Rare diseases collectively impact over 300 million people worldwide, yet for many, effective treatments remain elusive. With more than 7,000 identified rare diseases, the challenge is not only in developing targeted therapies but also in designing predictive preclinical models that can accurately mimic these complex conditions.

At MLM Medical Labs, we recognize that one-size-fits-all preclinical models don’t work when tackling rare diseases. We collaborate closely with sponsors to develop customized in vivo models, supporting drug discovery efforts from early research through regulatory submission. Our ability to offer both GLP and non-GLP studies, paired with comprehensive on-site ex vivo analyses, makes us a trusted partner for biotech and pharma companies striving to make an impact in the rare disease space.

The Challenge: Why Rare Disease Research Needs Specialized Preclinical Models

One of the biggest obstacles in rare disease drug development is the lack of established preclinical models. Unlike more common conditions such as obesity or psoriasis—where robust, standardized models exist—rare diseases often require highly specific, novel models.

Key Challenges in Rare Disease Research:

🔬 Limited Patient Populations – Small patient numbers make clinical trials difficult, increasing the importance of reliable preclinical models for regulatory approval.

🔬 Diverse Pathophysiology – Many rare diseases are genetically driven, requiring customized models that accurately reflect disease mechanisms.

🔬 Regulatory Hurdles – Demonstrating clinical relevance and efficacy to regulatory bodies is challenging without strong preclinical data.

🔬 High R&D Costs – Developing rare disease therapies is costly due to specialized research needs, complex biology, and limited market size.

Given these challenges, collaborating with a preclinical CRO that has the expertise to develop novel models is critical to ensuring a smooth path to clinical translation.

MLM Medical Labs: Your Partner in Rare Disease Preclinical Research

At MLM Medical Labs, we go beyond providing preclinical models—we custom-build them to align with specific disease mechanisms. Our approach is scientifically rigorous, integrating cutting-edge methodologies to ensure translational relevance.

Tailored Preclinical Models: Built for Innovation

At MLM Medical Labs, we don’t just provide models—we build them with you. Our preclinical experts work closely with sponsors to:

✅ Develop customized in vivo models based on specific inflammatory, metabolic, genetic or other rare disease mechanisms.

✅ Modify existing models to better replicate human disease phenotypes.

✅ Incorporate advanced biomarker analysis for precise disease tracking and therapeutic evaluation.

Whether it’s optimizing existing preclinical models or developing inflammation-based rare disease models, MLM’s preclinical team ensures that every model aligns with clinical relevance.

Seamless Transition from In Vivo to On-Site Ex Vivo Analysis

Whether it’s optimizing existing preclinical models or developing inflammation-based rare disease models, MLM’s preclinical team ensures that every model aligns with clinical relevance.

As part of our comprehensive research approach, we seamlessly integrate on-site ex vivo analysis to provide deeper insights into disease mechanisms and therapeutic effects. By leveraging advanced ex vivo methodologies, we ensure that preclinical findings translate effectively into clinical applications.

The Importance of Biomarker-Driven Preclinical Research

Biomarker analysis is a critical component in preclinical-to-clinical translation, enabling a deeper understanding of disease mechanisms, therapeutic efficacy, and patient stratification. Through precision-based approaches, biomarker-driven studies help identify early indicators of treatment response, allowing for more effective decision-making in drug development.

Our integrated biomarker-driven strategies enhance the predictability and relevance of preclinical models, ensuring they accurately mimic human disease progression. Our approach combines quantitative and qualitative data assessments, leveraging molecular profiling, tissue characterization, and immune response evaluation to refine therapeutic interventions. By incorporating modern technologies, customized biomarker panels and our scientific experts, we provide a more comprehensive understanding of drug efficacy and disease pathology.

Comprehensive On-Site Ex Vivo Analysis: Faster Insights, Greater Precision

One of the key advantages of working with MLM Medical Labs is our state-of-the-art in-house ex vivo capabilities, which eliminate the need for multiple vendors and ensure higher data integrity.

🔬 Biomarker Analysis – ELISA, multiplex cytokine profiling, qPCR, and more for disease monitoring and drug efficacy assessment.

🔬 Histopathology & Tissue Analysis – Advanced IHC, IF, and in situ hybridization for understanding cellular responses and disease progression.

🔬 Flow Cytometry & Immunophenotyping – Immune response profiling to assess therapeutic impact on disease-related pathways.

GLP & Non-GLP Studies: Designed for Every Stage

MLM Medical Labs offers both GLP-compliant and non-GLP study designs, allowing sponsors to:

🔹 Run early exploratory studies under non-GLP conditions for rapid proof of concept testing.

🔹 Generate robust regulatory data with GLP-compliant studies for IND-enabling submissions.

🔹 Maintain flexibility as programs evolve, seamlessly transitioning from non-GLP to GLP as needed.

With this adaptable approach, we support our clients at every step of drug development, from discovery through regulatory submission.

Bridging the Gap: From Preclinical Innovation to Clinical Impact

For rare disease patients and their families, every breakthrough matters. The journey from preclinical research to an approved therapy is long, complex, and uncertain, but with the right partners, the odds of success increase.

Our mission is to bridge the gap between scientific innovation and clinical application by providing:

✅ Custom model development to address the unmet needs of rare disease research.

✅ Regulatory-compliant studies that support IND and clinical trial applications.

✅ End-to-end analytical solutions for biomarker-driven drug development.

The Future of Rare Disease Research

Researching rare diseases is not just a scientific challenge—it is a humanitarian imperative. As we continue to deepen our understanding of rare disease mechanisms, we unlock new possibilities for targeted therapies, offering hope to millions of patients worldwide. Investing in rare disease research drives medical innovation, expands therapeutic frontiers, and paves the way for groundbreaking treatments that could transform the future of medicine. At MLM Medical Labs, we are excited to be at the forefront of these efforts, ensuring that scientific advancements translate into real-world impact for those who need it most.

Collaborate with MLM Medical Labs to Advance Rare Disease Therapeutics

Our integrated approach saves time, reduces variability, and provides decision-driving data to support faster preclinical development. With decades of experience in preclinical research, MLM Medical Labs is committed to helping biotech, pharmaceutical, and academic partners advance life-saving treatments for rare disease communities.

🚀 Are you working on a rare disease therapy? Let’s discuss how MLM Medical Labs can help accelerate your preclinical program and bring life-changing treatments to patients faster. Because Every Patient Deserves a Future—Accelerate Rare Disease Research Now CONTACT US today!